Designation granted by US FDA for the treatment of trigeminal neuralgia

Convergence Pharmaceuticals Limited ("Convergence" or "the company"), the company focused on the development of novel and high value analgesic medicines for the treatment of chronic pain, is pleased to announce that its novel sodium channel blocker, CNV1014802 ('802), has been granted orphan-drug designation by the US Food and Drug Administration for the treatment of trigeminal neuralgia (TN). Orphan-drug designation gives Convergence various incentives and advantages in the development of '802, including the potential availability of certain grants for clinical studies and assistance from the FDA with clinical protocol design. Furthermore orphan drug designation grants US market exclusivity, distinct from other types of exclusivity, for seven years if '802 is approved.

TN is a very severe form of facial pain that is experienced in short bursts or attacks called paroxysms. The International Association for the Study of Pain (IASP) defines TN as sudden, severe, brief, stabbing, recurrent episodes of pain usually on one side of the face and can be provoked by light touch. The pain follows one or more branches of the trigeminal nerve which provides nerve sensation from the mouth, face and the front of the scalp.

'802 is a novel small molecule that penetrates the central nervous system (CNS) and blocks Navs in a highly state-dependent fashion, while exhibiting selectivity against the 1.7 subtype. '802 is currently undergoing clinical evaluation in an innovative Phase II trial. An interim analysis from the trial reported in March that 70% of patients showed a successful response and were subsequently randomised into the double-blind treatment period. '802 was safe and well tolerated in these patients.

Clive Dix, Chief Executive Officer of Convergence Pharmaceuticals, commented: "This is a highly significant milestone for Convergence coming fast on the heels of positive interim data for '802. Trigeminal neuralgia is a highly debilitating disorder and current pain treatments come with a severe cost to quality of life. Orphan-drug designation will further smooth the path for the successful development of this novel sodium blocker and offer market exclusivity in the US if approved."

NOTES TO EDITORS

About Trigeminal Neuralgia (TN)

TN currently affects approximately 50,000 people in the USA alone. The majority of people affected are over 50 years of age; however, many cases have been reported in young adults. TN is more prevalent in women than men, and for most sufferers, the condition is progressive and worsens over time. TN is commonly misdiagnosed and to date there is no guaranteed cure for the condition. Current therapies are centred on sodium channel blockers such as carbamazepine or oxcarbazepine as first-line treatments. However, these agents, although providing relief, are often poorly tolerated, and require lengthy dose escalation, resulting in sub-optimal efficacy.

About the pain therapeutic area

The pain therapeutic area encompasses any disease where pain is a major symptom. The unmet medical need for patients in pain is enormous, with the greatest need being for a more effective therapy that is well tolerated and safe over a long period of time. Within the pain marketplace, current pain treatments are unsatisfactory; overall efficacy is poor (typically 1-2 point reductions on a 10 point scale) and satisfactory to less than 50% of patients. Whilst treatments, such as opioids and non-steroidal anti-inflammatory drugs, are available on the market, many patients obtain little or no relief from these existing analgesics and often such drugs are associated with adverse events, side effects and addiction concerns. This highlights the importance of Convergence Pharmaceuticals' goals to be a world leader in the development of novel, efficacious analgesics with improved pharmacology and safety profiles.

About chronic pain

Currently, more than 1.5 billion people worldwide suffer from chronic pain of varying degrees. Among all types of chronic pain, neuropathic pain stands out with approximately 3-4.5% of the global population affected, with incidence rate increasing in line with increased age of the population. With the unmet clinical need so high, the demand for better pain management therapies, addressing acute and chronic pain, is on the rise.

According to recent research the global pain management market is to reach US$60 Billion by 2015.[1]

Convergence Pharmaceuticals is an independent biotechnology company focused on the development of novel analgesics with potentially commercially attractive efficacy, responder-rate and side effect profiles. The Company, led by CEO Clive Dix, was formed in October 2010 following the acquisition of certain neuroscience clinical assets from GlaxoSmithKline ("GSK"). The Company has a pipeline of differentiated clinical-stage compounds targeting the points of convergence in chronic pain signalling through modulation of specific ion-channels.

Convergence Pharmaceuticals is well funded and raised US$35.4 million in Series A financing from a syndicate of leading European and US financial institutions. For more information please go to the Company's website at http://www.convergencepharma.com

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